Belzutifan for HIF2A-Related Pheochromocytoma and Paraganglioma: A Retrospective Study of Real-World Data
Authors' conclusion
Belzutifan is a highly effective and well-tolerated therapeutic option for EPAS1(HIF2A)-related PPGL, producing substantial biochemical and radiographic responses, improved blood pressure control, and resolution of erythrocytosis. These findings support belzutifan as a promising genotype-driven therapy for pseudohypoxia-driven PPGL.
Study context
- Population
- Patients with recurrent, multifocal or metastatic EPAS1 (HIF2A)-related pheochromocytoma and paraganglioma (PPGL)
- Participants
- 5 patients
- Outcome
- Tumor response (RECIST 1.1)
Methodological detailsreal-world data
- Real-world data
Main finding
As reported in the abstract
Partial response
4 of 5 patients
80%
Stable disease
1 patient
Average reduction in sum of tumor diameters
36.8%
Observational data: associations, not effects.
Further findings
Biochemical markers
- Decline in chromogranin A
- 69%
- Decline in erythropoietin
- 97%
- Decline in hemoglobin
- 13%
- Need for therapeutic phlebotomy eliminated in patients with prior erythrocytosis
Antihypertensive medications
- Catecholamine normalization allowed discontinuation or reduction of antihypertensives
- 2 patients
Safety
Adverse events
- Hypoxia and anemia
- 1/5
- Mild transaminase elevation
- 2/5
- Fatigue with weight gain
- 1/5
Original publication
Belzutifan for HIF2A-Related Pheochromocytoma and Paraganglioma: A Retrospective Study of Real-World Data. Endocr Pract. Published 1 October 2025. doi:10.
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